EP4061833A4 - Compositions and methods for derepressing re1 silencing transcription factor target genes - Google Patents

Compositions and methods for derepressing re1 silencing transcription factor target genes Download PDF

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Publication number
EP4061833A4
EP4061833A4 EP20890436.7A EP20890436A EP4061833A4 EP 4061833 A4 EP4061833 A4 EP 4061833A4 EP 20890436 A EP20890436 A EP 20890436A EP 4061833 A4 EP4061833 A4 EP 4061833A4
Authority
EP
European Patent Office
Prior art keywords
derepressing
compositions
methods
transcription factor
target genes
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP20890436.7A
Other languages
German (de)
French (fr)
Other versions
EP4061833A1 (en
Inventor
Edmund NESTI
Alexander Pisarchik
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Alcamen Stem Cell Therapeutics LLC
Original Assignee
Alcamen Stem Cell Therapeutics LLC
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Alcamen Stem Cell Therapeutics LLC filed Critical Alcamen Stem Cell Therapeutics LLC
Publication of EP4061833A1 publication Critical patent/EP4061833A1/en
Publication of EP4061833A4 publication Critical patent/EP4061833A4/en
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/08—Linear peptides containing only normal peptide links having 12 to 20 amino acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03016—Phosphoprotein phosphatase (3.1.3.16), i.e. calcineurin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
EP20890436.7A 2019-11-22 2020-11-20 Compositions and methods for derepressing re1 silencing transcription factor target genes Pending EP4061833A4 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962939149P 2019-11-22 2019-11-22
US202063086248P 2020-10-01 2020-10-01
PCT/US2020/061632 WO2021102345A1 (en) 2019-11-22 2020-11-20 Compositions and methods for derepressing re1 silencing transcription factor target genes

Publications (2)

Publication Number Publication Date
EP4061833A1 EP4061833A1 (en) 2022-09-28
EP4061833A4 true EP4061833A4 (en) 2023-09-06

Family

ID=75981713

Family Applications (1)

Application Number Title Priority Date Filing Date
EP20890436.7A Pending EP4061833A4 (en) 2019-11-22 2020-11-20 Compositions and methods for derepressing re1 silencing transcription factor target genes

Country Status (11)

Country Link
US (1) US20230059411A1 (en)
EP (1) EP4061833A4 (en)
JP (1) JP2023503920A (en)
KR (1) KR20220104008A (en)
CN (1) CN115023434A (en)
AU (1) AU2020386637A1 (en)
BR (1) BR112022009898A2 (en)
CA (1) CA3162241A1 (en)
IL (1) IL293165A (en)
MX (1) MX2022006214A (en)
WO (1) WO2021102345A1 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115887655A (en) * 2021-09-30 2023-04-04 中国科学院脑科学与智能技术卓越创新中心 Direct transdifferentiation treatment of neurological disorders
CN114377000A (en) * 2021-12-24 2022-04-22 中山大学孙逸仙纪念医院 Application of pregabalin in preparation of medicine for preventing or treating radioactive brain injury

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150299698A1 (en) * 2012-11-01 2015-10-22 The Regents Of The University Of California Methods for engineering non-neuronal cells into neurons and using newly engineered neurons to treat neurodegenerative diseases
WO2016019315A2 (en) * 2014-08-01 2016-02-04 Oregon Health & Science University Methods and compositions useful in manipulating the stability of re1 silencing transcription factor

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US2019A (en) 1841-03-29 goedes
JP2012525146A (en) * 2009-04-28 2012-10-22 プレジデント アンド フェロウズ オブ ハーバード カレッジ Overcharged protein for cell penetration
EP2550356A4 (en) * 2010-03-25 2013-09-25 Int Stem Cell Corp Method of altering the differentiative state of a cell and compositions thereof

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150299698A1 (en) * 2012-11-01 2015-10-22 The Regents Of The University Of California Methods for engineering non-neuronal cells into neurons and using newly engineered neurons to treat neurodegenerative diseases
WO2016019315A2 (en) * 2014-08-01 2016-02-04 Oregon Health & Science University Methods and compositions useful in manipulating the stability of re1 silencing transcription factor

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
BURKHOLDER NATHANIEL TATE ET AL: "Phosphatase activity of small C-terminal domain phosphatase 1 (SCP1) controls the stability of the key neuronal regulator RE1-silencing transcription factor (REST)", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 293, no. 43, 1 October 2018 (2018-10-01), US, pages 16851 - 16861, XP093067466, ISSN: 0021-9258, DOI: 10.1074/jbc.RA118.004722 *
CHEN ZAN ET AL: "Synthetic approaches to protein phosphorylation", CURRENT OPINION IN CHEMICAL BIOLOGY, vol. 28, 1 October 2015 (2015-10-01), GB, pages 115 - 122, XP093067587, ISSN: 1367-5931, DOI: 10.1016/j.cbpa.2015.07.001 *
DANA MARIA COPOLOVICI ET AL: "Cell-Penetrating Peptides: Design, Synthesis, and Applications", ACS NANO, vol. 8, no. 3, 24 February 2014 (2014-02-24), US, pages 1972 - 1994, XP055568279, ISSN: 1936-0851, DOI: 10.1021/nn4057269 *
See also references of WO2021102345A1 *

Also Published As

Publication number Publication date
AU2020386637A1 (en) 2022-06-16
MX2022006214A (en) 2022-06-22
CA3162241A1 (en) 2021-05-14
WO2021102345A1 (en) 2021-05-27
EP4061833A1 (en) 2022-09-28
JP2023503920A (en) 2023-02-01
BR112022009898A2 (en) 2022-09-06
US20230059411A1 (en) 2023-02-23
IL293165A (en) 2022-07-01
KR20220104008A (en) 2022-07-25
CN115023434A (en) 2022-09-06

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